Profile

Arnaud Klein

Research scientist

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Titles and positions

  • Research scientist  INSERM
  • PhD in Molecular biology (Université de Montréal, Canada)

Expertises

  • Myotonic Dystrophy
  • Cell and Molecular biology
  • Alternative splicing
  • Therapeutic approaches

Presentation

Researcher in team 4/ Furling group, my projects mainly focus on the development of innovative therapeutic strategies for Myotonic Dystrophy type 1. We use gene therapy approaches or antisense oligonucleotides that we evaluate in cellular (myoblasts, fibroblasts or overexpression) and mouse models of the disease.

Main publications

  1. Klein, AF, Arandel, L, Marie, J, Furling, D. FISH Protocol for Myotonic Dystrophy Type 1 Cells. Methods Mol Biol. 2020;2056 :203-215. doi: 10.1007/978-1-4939-9784-8_13. PubMed PMID:31586350 .
  2. Klein, AF, Varela, MA, Arandel, L, Holland, A, Naouar, N, Arzumanov, A et al.. Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice. J Clin Invest. 2019;129 (11):4739-4744. doi: 10.1172/JCI128205. PubMed PMID:31479430 PubMed Central PMC6819114.
  3. Matloka, M, Klein, AF, Rau, F, Furling, D. Cells of Matter-In Vitro Models for Myotonic Dystrophy. Front Neurol. 2018;9 :361. doi: 10.3389/fneur.2018.00361. PubMed PMID:29875732 PubMed Central PMC5974047.
  4. Arandel, L, Polay Espinoza, M, Matloka, M, Bazinet, A, De Dea Diniz, D, Naouar, N et al.. Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds. Dis Model Mech. 2017;10 (4):487-497. doi: 10.1242/dmm.027367. PubMed PMID:28188264 PubMed Central PMC5399563.
  5. Laurent, FX, Sureau, A, Klein, AF, Trouslard, F, Gasnier, E, Furling, D et al.. New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeats. Nucleic Acids Res. 2012;40 (7):3159-71. doi: 10.1093/nar/gkr1228. PubMed PMID:22156369 PubMed Central PMC3326330.
  6. Fugier, C, Klein, AF, Hammer, C, Vassilopoulos, S, Ivarsson, Y, Toussaint, A et al.. Misregulated alternative splicing of BIN1 is associated with T tubule alterations and muscle weakness in myotonic dystrophy. Nat Med. 2011;17 (6):720-5. doi: 10.1038/nm.2374. PubMed PMID:21623381 .
  7. François, V, Klein, AF, Beley, C, Jollet, A, Lemercier, C, Garcia, L et al.. Selective silencing of mutated mRNAs in DM1 by using modified hU7-snRNAs. Nat Struct Mol Biol. 2011;18 (1):85-7. doi: 10.1038/nsmb.1958. PubMed PMID:21186365 .
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